Clinical Trial: Efficacy and Safety Study of Allogenic Mesenchymal Stem Cells for Patients With Chronic Graft Versus Host Disease

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

Official Title: Phase Ⅱ/Ⅲ Clinical Trial, Multicenter, Randomized, Controlled, for the Evaluation of Efficacy and Safety of Therapy With Allogenic Mesenchymal Stem Cells in Patients With The primary purpose of the study is to evaluate the safety and efficacy of mesenchymal stem cells (MSC) for the treatment of patients who have developed an extensive chronic graft versus host disease (with skin and/or liver damage) after HSCs transplantation and do not respond to first-line therapy.

The secondary purpose of the study is to evaluate the effect of mesenchymal stem cells (MSC) on one-year survival rate, long-term survival rate, life quality and recurrence of patients who have developed an extensive chronic graft versus host disease (with skin and/or liver damage) after HSCs transplantation and do not respond to hormone treatment.


Detailed Summary: Chronic Graft-versus-host disease (GVHD)， with the incidence of 30%-60%, is a serious late complication of allogeneic hematopoietic stem cell transplantation (HSCT) and is the major cause of death in the late stage of transplantation. According to targeted organs, cGVHD is divided into two types, limited cGVHD and extensive cGVHD. Extensive cGVHD needs systemic immunosuppressant treatment. However, currently standard first-line regimen including cyclophosphamide and prednisolone is only effective for some patients. Novel treatment is urgently needed. Our previous study has shown that mesenchymal stem cells (MSCs) are effective for cGVHD patients with multiple skin damage. To further explore the therapeutic effect of MSCs for extensive cGVHD, we plan to conduct a multi-center clinical trial. Patients who developed an extensive cGVHD (with skin and/or liver damage) after HSCs transplantation and do not respond to first-line therapy are enrolled. They will be randomly divided into two groups which will receive MSCs and routine second-line drugs respectively. We will evaluate the efficacy and safety of MSCs for extensive cGVHD by comparison of symptom improvement, survival rate, recurrence as well as side effects in the two groups.
Sponsor: Chinese Academy of Medical Sciences

Current Primary Outcome: The total Response rate defined as patients with complete and partial response [ Time Frame: 1 year after MSCs administration. ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • one-year survival rate [ Time Frame: 1 year after MSCs administration ]
  • disease relapse [ Time Frame: 2 years after MSCs administration ]
  • quality of life [ Time Frame: 2 years after MSCs administration ]


Original Secondary Outcome: Same as current

Information By: Chinese Academy of Medical Sciences

Dates:
Date Received: February 1, 2012
Date Started: February 2012
Date Completion: June 2014
Last Updated: August 1, 2012
Last Verified: February 2012